FDA grants Orphan Drug status to uniQure's Fabry therapy

LEXINGTON, Mass. and AMSTERDAM - uniQure N.V. (NASDAQ: NASDAQ:QURE), a gene therapy company, announced today that the U.S. Food and Drug Administration (FDA) has awarded Orphan Drug Designation to its investigational gene therapy AMT-191 for Fabry disease treatment. This designation is intended to advance the development of treatments for rare diseases affecting fewer than 200,000 people in the U.S., providing various incentives such as tax credits and market exclusivity post-approval.

Fabry disease is a genetic condition characterized by the buildup of lipids in various cells, which can lead to severe organ damage. The current standard of care involves bi-weekly enzyme replacement infusions, which may not be fully effective for all patients.

AMT-191, a one-time intravenous therapy, aims to deliver a functional copy of the GLA gene to the liver to produce the missing enzyme in Fabry patients. The Phase I/IIa clinical trial, which began dosing patients in August 2024, will evaluate the safety, tolerability, and initial efficacy of two dosage levels in adult male participants over a two-year period.

Dr. Walid Abi-Saab, chief medical officer of uniQure, emphasized the importance of developing new gene therapies given the limitations of existing treatments. The company expects to provide initial clinical data from the trial in 2025.

The FDA's Orphan Drug Designation underscores the urgent need for innovative therapeutic options for rare diseases and supports the ongoing clinical trial of AMT-191. uniQure has previously achieved a significant milestone with the approval of its gene therapy for hemophilia B and is continuing to develop gene therapies for a range of severe diseases.

This news is based on a press release statement from uniQure Inc.

In other recent news, uniQure N.V. continues to make strides in the gene therapy sector. The company has reported encouraging interim data from Phase I/II trials of AMT-130, a gene therapy candidate for Huntington's disease. The trials have shown a significant slowing in disease progression and a notable reduction in neurofilament light protein, a neurodegeneration marker. H.C. Wainwright and Stifel have maintained a Buy rating on uniQure, following these positive trial updates and the FDA's granting of an RMAT designation to AMT-130.

Additionally, the company has initiated a significant organizational restructuring, including a 65% reduction in its workforce and the sale of its manufacturing facility in Lexington, Massachusetts. This move is part of uniQure's strategy to prioritize the development of its gene therapy programs.

In a major development, Genezen has acquired uniQure's commercial gene therapy operations in Lexington, enhancing its global gene therapy development capabilities. The Lexington site will serve as Genezen's global AAV center of excellence.

On the governance side, uniQure's shareholders have approved an expansion of the company's share incentive plan, reappointment of board members, and the adoption of the 2023 statutory annual accounts. However, Paula Soteropoulos, a non-executive director, has chosen not to stand for re-election at the next annual meeting. These are the recent developments in the company's operations.

InvestingPro Insights

As uniQure N.V. (NASDAQ: QURE) continues to make strides in the field of gene therapy with its recent FDA Orphan Drug Designation for AMT-191, investors are closely monitoring the company's financial health and market potential. According to real-time data from InvestingPro, uniQure's market capitalization stands at a modest $261.02 million, reflecting the size of the company within the biotechnology space. Despite the challenges faced by the company, including a significant revenue decline over the last twelve months of -75.25%, there is a notable quarterly revenue growth of 359.37% as of Q2 2024, indicating potential signs of a turnaround or milestone achievements.

InvestingPro Tips highlight that analysts are expecting sales growth in the current year, which could be a positive indicator for the company's future performance. Additionally, the fact that 6 analysts have revised their earnings upwards for the upcoming period suggests growing optimism about uniQure's prospects. This is particularly relevant given the company's focus on developing groundbreaking treatments like AMT-191 for Fabry disease. On the flip side, analysts do not anticipate the company will be profitable this year, and uniQure has been quickly burning through cash, which could raise concerns about long-term financial sustainability.

For investors seeking more detailed analysis and additional insights, there are 7 more InvestingPro Tips available for uniQure at https://www.investing.com/pro/QURE. These tips provide a deeper dive into the company's financials and market position, offering valuable information for making informed investment decisions.

InvestingPro's fair value estimate for uniQure is currently at $6.59, which is above the previous close price of $5.36. This suggests that the stock might be undervalued at its current trading price, presenting a potential opportunity for investors. However, with any investment, especially in the high-risk biotech sector, it is crucial to consider the full picture, including both the potential rewards and risks involved.

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